Drug Delivery

Image from NIEHS, NIHAdvances in cellular and molecular biology, together with an increased understanding of Parkinson's, have led to new approaches to modifying the course of the disease. These include the development of small molecules against a particular receptor, large biologics (such as neurotrophic factors) that demonstrate neuroprotection in pre-clinical models, and genetic material such as siRNA or or adeno- or lentiviral-encapsulated genes that target a particular gene or gene product. Researchers are currently forced to optimize the size and composition of drugs to get them across the blood-brain barrier in sufficient amounts. Often this optimization results in loss of drug potency or efficacy. Eventually, each therapeutic molecule faces the same challenge: successful delivery to specific parts of the brain, at appropriate levels, in a controlled and reproducible fashion.

Two main approaches are being investigated to overcome the issue of drug delivery in CNS disorders, including PD. First, technologies that improve delivery of therapeutics across the blood-brain barrier are essential for non-invasive approaches. Second, direct brain infusion or implantation using surgical approaches can be used to deliver the therapeutic molecule to the brain region of interest.